Taking on a Critical Role in Addressing Unmet Medical Needs

Healthy volunteers and rare disease patients participate in important research.

By Tim Green
01 Mar 2018

Discovering and developing a new medicine is an expensive and time-consuming business. It is estimated that the average cost of a new, registered medicine is around $2.6 billion, with the average time from discovery to launch being more than 10 years.

In order to mitigate the risk of investing in an ineffective therapy, biopharmaceutical companies apply rigorous testing mechanisms that provide early feedback about whether or not to proceed with their development.

These mechanisms operate throughout the R&D life-cycle of a candidate drug; from initial concept through pre-clinical and clinical research of molecules, right through to final product registration and launch.

Global biotechnology leader CSL carefully selects and rigorously tests new therapies in the development of a new generation of high tech medicines based on recombinant protein technologies.

In collaboration with colleagues around the world, CSL research scientists develop biological tests (bioassays) that are designed to evaluate the effectiveness and mechanism of action of new drugs.

These bioassays often use cells separated from human blood to demonstrate whether the investigational medicine is targeting and inhibiting the right molecules or pathways that are believed to be driving a particular disease process. The tests provide information about the safety, efficacy and potency of the candidate drug.

In order to develop these bioassays, researchers require blood samples from patients with relevant diseases as well as blood from healthy volunteers. Blood from healthy volunteers is critical to the research, enabling rigorous scientific comparison between the effects of the candidate medicine on disease processes compared with normal biological mechanisms.

To facilitate these comparison tests, CSL scientists have been working with the online patient recruitment service, Clinical Trials Connect to source blood for medical research from volunteers attending the Skin and Cancer Foundation Clinic in Carlton, Australia.

The donation process, which is approved by an independent ethics committee under the terms of the Human Tissue Act (Vic), is carefully managed to ensure donors are fully informed about what their blood will be used for.

Once collected, the blood is transported to research labs and is usually used the same day in bioassays for drug development. Occasionally the blood is processed in a way that allows it to be frozen for use at a later time.

During the past five years, CSL has utilized donations of blood samples from more than 900 volunteers. These donations have been used to support research and development of new medicines designed to treat cancer, cardiovascular, metabolic, inflammatory and immune deficiency related diseases.

CSL researchers are grateful for the support provided by our research partners and the volunteers whose blood samples have enabled significant progress to be made in the development of a number of new medicines.